Technology ID

Mouse Models of Cryopyrin-Associated Periodic Syndrome (CAPS) for Drug Discovery

Lead Inventor
Brydges, Susannah (Regeneron Pharmaceuticals, Inc)
Hoffman, Harold (University of California, San Diego)
Kastner, Daniel
Mueller, James (University of California, San Francisco (UCSF))
Research Materials
Therapeutic Areas
Development Stages
Pre-clinical (in vivo)
Research Products
Animal Models
Lead IC

This technology includes mouse models that express versions of mouse cryopyrin protein containing mutations associated with human CAPS disease. We engineered mutations associated with three specific CAPS phenotypes (familial cold autoinflammatory syndrome (FCAS); Muckle-Wells syndrome (MWS); and neonatal onset multisystem inflammatory disease (NOMID)) into the mouse cryopyrin gene (called Nlrp3) to examine the roles of IL-1 β and related cytokines, and better characterize inflammasome functions.

Commercial Applications
Mice could be used for testing additional IL-1, IL-18, and/or inflammasome or innate immune targeting drug moieties before entering the clinic.

Competitive Advantages
First available mouse model of CAPS and only available model of uncontrolled IL-1β signaling (the IL-1 receptor antagonist knock-out) did not address IL-1 β independent functions of the inflammasome.
Licensing Contact:
Knezevic, Vladimir